251. 尿素サイクル異常症 Urea cycle disorder Clinical trials / Disease details
臨床試験数 : 54 / 薬物数 : 61 - (DrugBank : 15) / 標的遺伝子数 : 3 - 標的パスウェイ数 : 28
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | EUCTR2015-000988-14-Outside-EU/EEA (EUCTR) | 12/10/2016 | Human Heterologous Liver Cells for Infusion in Children With Urea Cycle Disorders | Title: Open, Prospective, Historic-Controlled, Multicenter Study toEvaluate the Safety and Efficacy of Infusion of Liver Cell Suspension(HHLivC) in Children with Urea Cycle Disorders | Neonatal-onset type of the following urea cycle disorders(UCD):ornithine transcarbamylase deficiency (OTCD),carbamoylphosphate synthetase I deficiency (CPS1D), orargininosuccinate synthetase deficiency (ASSD orcitrullinemia);Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: Heparesc Product Code: HHLivC INN or Proposed INN: not available Other descriptive name: ALLOGENIC HUMAN HETEROLOGOUS LIVER CELLS | PROMETHERA Biosciences S.A./N.V. | NULL | NA | Female: yes Male: yes | 21 | Phase 2 | United States;Canada |